ELBIO Inc invented an osmotic polymer-based gene delivery system and 1. secured proprietary platform technology. Using this platform, the company 1. developed cancer cell-targeted siRNA gene therapeutics and obtained patents 1. in Korea, the United States (October 2018), and the European Union 1. including three European countries (October 2023).

Using the same gene delivery platform, ELBIO developed a novel gene anticancer therapeutic in which a cancer stem cell-targeting peptide is conjugated to the delivery carrier and loaded with siRNA or CRISPR designed to block the self-renewal of cancer stem cells, thereby inducing their self-destruction. Patents for this delivery system and gene therapeutics were registered in Korea (2022), China (2024), and Japan (2024), while patent applications are currently under examination in the United States and India.

When cancer cell-killing siRNA nano-anticancer therapeutics were applied to an orthotopic mouse glioblastoma (GBM) model, intravenous (IV) treatment administered every three days for two weeks reduced tumor volume by approximately 65%. Furthermore, a nanochain-formulated siRNA anticancer therapeutic demonstrated enhanced efficacy, reducing tumor volume by approximately 87% following two weeks of IV treatment.

When cancer cell-killing siRNA anticancer therapeutics were applied to a mouse subcutaneous xenograft model using human lung cancer cells (A549), intratumoral (IT) treatment administered every three days for 30 days reduced tumor volume by approximately 80%.

When cancer cell-killing siRNA anticancer therapeutics were applied to pancreatic cancer cells (BxPC-3), approximately 30% cell death was observed following a single treatment in vitro. In an in vivo orthotopic mouse pancreatic cancer model, intraperitoneal (IP) administration performed five times at three-day intervals over 12 days resulted in approximately 70% reduction in tumor weight when evaluated 15 days later, demonstrating strong therapeutic efficacy.

Since siRNA anticancer therapeutics demonstrated therapeutic efficacy in mouse models of brain cancer, lung cancer, and pancreatic cancer, GLP-compliant nonclinical studies including pharmacokinetic (PK) studies, toxicokinetic (TK) toxicity studies in mice and non-human primates, as well as efficacy evaluation studies in mice, are being planned.